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Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium. 2020
Debra A Thompson, and
Alessandro Iannaccone, and
Robin R Ali, and
Vadim Y Arshavsky, and
Isabelle Audo, and
James W B Bainbridge, and
Cagri G Besirli, and
David G Birch, and
Kari E Branham, and
Artur V Cideciyan, and
Steven P Daiger, and
Deniz Dalkara, and
Jacque L Duncan, and
Abigail T Fahim, and
John G Flannery, and
Roberto Gattegna, and
John R Heckenlively, and
Elise Heon, and
K Thiran Jayasundera, and
Naheed W Khan, and
Henry Klassen, and
Bart P Leroy, and
Robert S Molday, and
David C Musch, and
Mark E Pennesi, and
Simon M Petersen-Jones, and
Eric A Pierce, and
Rajesh C Rao, and
Thomas A Reh, and
Jose A Sahel, and
Dror Sharon, and
Paul A Sieving, and
Enrica Strettoi, and
Paul Yang, and
David N Zacks, and
Department of Ophthalmology and Visual Sciences, Kellogg Eye Center, University of Michigan Medical School, Ann Arbor, MI, USA.
Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to develop treatments for these blinding conditions at the forefront of the emerging field of precision medicine. As a result, the growth of clinical trials for IRDs has increased rapidly over the past decade and is expected to further accelerate as more therapeutic possibilities emerge and qualified participants are identified. Although guided by established principles, these specialized trials, requiring analysis of novel outcome measures and endpoints in small patient populations, present multiple challenges relative to study design and ethical considerations. This position paper reviews recent accomplishments and existing challenges in clinical trials for IRDs and presents a set of recommendations aimed at rapidly advancing future progress. The goal is to stimulate discussions among researchers, funding agencies, industry, and policy makers that will further the design, conduct, and analysis of clinical trials needed to accelerate the approval of effective treatments for IRDs, while promoting advocacy and ensuring patient safety.
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